News briefing: Pfizer on track to early '22 pivotal readout on hemophilia A; Codiak scales back IPO expectations
Pfizer and Sangamo have dosed their first patient in the Phase III gene therapy trial of giroctocogene fitelparvovec (SB-525) for hemophilia A. Researchers will be following the experiences of patients for 12 months after dose, checking on safety, efficacy and durability. The move comes on the heels of a major setback at rival BioMarin, which received a rejection letter from the FDA, which demanded more proof of durability — a sore point among some of the analysts tracking the program.
A spokesperson for Pfizer tells Endpoints News today that they plan “a pivotal readout in early 2022 for giroctocogene fitelparvovec.”
Brenda Cooperstone, Pfizer’s chief development officer, rare disease, noted: “Enrollment in the lead-in study is progressing well and recruitment is on track for Phase 3. Given the Phase 1/2 study findings to date, we believe that giroctocogene fitelparvovec has the potential to sustain factor levels and reduce annual bleed rates, suggesting this one-time gene therapy could potentially transform the standard of care for eligible patients worldwide.” — John Carroll
Codiak scales down IPO expectations to $83M
Amid a sizzling hot biotech IPO environment where most companies are bringing in more than they initially planned, Codiak BioSciences is tuning down expectations as it sets terms.
Having penciled in $100 million in their IPO filing, the exosome specialists now say they plan to raise $83 million by offering 5.5 million shares at a price range of $14 to $16. That’s lower than what they would have raised in 2019, when they launched but ultimately pulled an attempt to go public due to unfavorable market conditions.
At the midpoint of the proposed range, Codiak would command a fully diluted market value of $309 million, according to Renaissance Capital.
A pioneer in co-opting exosomes, the body’s natural intercellular communication system, to deliver therapeutic payloads to specific addresses, Codiak’s lead program is designed to activate the STING pathway to treat solid tumors. It expects to start Phase I/II later this year and reap preliminary data by mid-2021. — Amber Tong
Purdue reportedly nearing guilty plea in federal opioid probe
Purdue Pharma is nearing a plea deal where it would plead guilty to criminal charges related to its role in aggressively marketing highly addictive opioids, according to a Reuters report.
Run by the Sackler family, Purdue would reportedly face fines exceeding $8 billion should the agreement be reached. Those fines include about $3.54 billion in a criminal fine, a $2 billion criminal forfeiture and a $2.8 billion civil penalty, per Reuters.
The agreement could be reached sometime within the next two weeks. Because of Purdue’s bankruptcy proceedings, the fines are unlikely to be paid out anytime soon given that the company lacks the cash at present to repay all creditors.
Speaking to Reuters, a Justice Department spokesperson declined to comment but said that the report “contains inaccuracies and is highly misleading” without explaining.
Prosecutors across the country, at the federal and state levels, have alleged Purdue made billions of dollars in profits from its opioids and sent illegal kickbacks to doctors and pharmacies. Purdue filed for bankruptcy last year following several lawsuits and criminal cases over its role in fueling the opioid crisis. — Max Gelman
Abeona forms special committee to oversee leadership
Following a rough few years, Abeona Therapeutics announced the formation of a special committee of board members to oversee its executive leadership.
In addition, the company has brought in Jefferies to conduct a review of strategic operations. The company said there’s no defined timeline for the review, and that it doesn’t intend to comment further unless “a specific initiative is approved by the board of directors, the review process is concluded, or it is otherwise determined that other disclosure is appropriate.”
Back in 2018, Abeona chief Carsten Thiel was ousted following accusations of unspecified “personal misconduct.” And the following year, the FDA pumped the brakes on the biotech’s Phase III study of its potential treatment for recessive dystrophic epidermolysis bullosa. The FDA lifted its hold in December, allowing Abeona to resume testing.
Last October, Abeona’s $ABEO shares were at $2.38. On Tuesday, they closed at $1.22.
The special committee will work “closely with the executive leadership team to develop the company’s strategic direction and leadership plan,” Abeona announced. — Nicole DeFeudis
Urovant and Sunovion strike marketing deal for potential overactive bladder drug
Urovant has struck a 5-year deal with Sunovion Pharmaceuticals to market its overactive bladder drug vibegron in primary care in the US, should it get approval in December.
The FDA accepted Urovant’s NDA for vibegron in March, and set a PDUFA date for Dec 26. Urovant in-licensed the drug from Merck with a $25 million upfront. Topline Phase III data showed that it hit statistical significance for both co-primary endpoints and all 7 secondaries when compared against a placebo.
According to the company, it’s also being evaluated for OAB in men with benign prostatic hyperplasia, and for abdominal pain associated with irritable bowel syndrome.
“While Urovant sales representatives will be focused on urologists, long-term care, and high-prescribing PCPs, the Sunovion team will significantly broaden our reach into the U.S. primary care community,” Urovant’s senior commercial VP Walt Johnston said in a statement.
The new deal is in addition to an exclusive 3-year agreement the companies reached in June for wholesale trade and retail distribution, contract operations and select account management for vibegron. — Nicole DeFeudis